Israeli stem cell therapeutics company BrainStorm Cell Therapeutics Ltd. (Bulletin Board:BCLI), which develops adult stem cell therapeutics for neurodegenerative diseases, has announced success in a clinical trial on ALS patients. 36 patients treated with the company's stem cell treatment NurOwn exhibited slower degeneration than the twelve patients in the control group. "The study achieved its primary objective, demonstrating that NurOwn was safe and well tolerated. NurOwn also achieved multiple secondary efficacy endpoints, showing clear evidence of a clinically meaningful benefit. Notably, response rates were higher for NurOwn-treated subjects compared to placebo at all time points in the study out to 24 weeks," the company said.
The results enable the company to continue its clinical trials. In the next trial patients will be treated several times and not only once. It is still not known whether the next trial will be larger than the current one, and whether it will be defined as a Phase 3 trial or an additional Phase 2 trial.
BrainStorm's share price spiked on Friday to $3.64 but subsided to $2.71 by yesterday's close, giving the company a market cap of $55.5 million.
BrainStorm's treatment uses cells taken from the patient's own bone marrow and processed using the company's unique technology that are then replanted in the patient's spine.
Because of the small number of patients in the control group, the trial cannot be declared as clearly demonstrating an advantage in using the treatment, but there is substantial indication of its efficacy.
"We are very encouraged by these Phase 2 data that show that NurOwn cell therapy is safe and that a single administration produces a transient and clinically meaningful beneficial response in terms of both the ALS-FRS-R rating scale and CSF biomarkers," said Dr. Robert H. Brown, Professor and Chair of Neurology at the University of Massachusetts Medical Center and Medical School, and investigator in the trial, "These exciting findings clearly indicate that it is appropriate to conduct a longer study with repetitive dosing."
The trial was conducted at the University of Massachusetts Medical Center, the Massachusetts General Hospital, and the Mayo Clinic in Minnesota.
Chaim Lebovits, CEO of BrainStorm, said, "This study met its objectives, demonstrating both the safety of NurOwn and its ability to provide clinical benefit to ALS patients, and most importantly, will help us to determine the study population and design for a pivotal study of multiple doses of NurOwn in ALS."
Published by Globes [online], Israel business news - www.globes-online.com - on July 19, 2016
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